20.500.12556/DiRROS-24945 Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy real-world criteria for transition to vamorolone therapy Aims: To assess the prevalence of endocrine/metabolic disorders among pediatric Duchenne muscular dystrophy (DMD) patients and identify individuals who would benefit from the new corticosteroid treatment available, vamorolone. Methods: A national pediatric cohort of DMD patients followed at University Children’s Hospital, University Medical Center Ljubljana in June 2025 was included in the study. The presence of endocrine/metabolic disorders was determined by clinical examination, laboratory data, and imaging at the last annual multidisciplinary evaluation. Results: A total of 21 patients (average age 11.1, range 4.6-16.7 yrs) were included in the study. Two patients were corticosteroid-naive, the rest treated with deflazacort (average treatment duration 5.4, range 1.1-11.3 yrs). At the time of final follow-up, 90% were diagnosed with an endocrine and/or metabolic disorder. Short stature was present in 67% of patients, a decline in height standard deviation score (SDS) since the initiation of corticosteroid therapy was observed, -2.37 SDS on average. Based on body composition data (fat % SDS), 57% were classified as obese. Lipid abnormalities were detected in 76% of patients. Insulin resistance (determined by HOMA-IR) was increased in 9.5% of patients; type 2 diabetes was not detected. Conclusion: Our findings demonstrate a high prevalence of endocrine and metabolic disturbances among patients with DMD. In light of emerging evidence on the potential benefits of vamorolone - particularly regarding bone health and growth - we identified a subgroup of patients most likely to benefit from its use. We propose that ambulatory, non-corticosteroid naive patients with pathological fractures, markedly reduced bone density, short stature, or significant growth deceleration could be considered for the therapeutic transition. Accordingly, we established national clinical criteria to guide individualized therapeutic transitions, aimed at optimizing clinical outcomes and ensuring efficient allocation of healthcare resources. Duchenne muscular dystrophy corticosteroid treatment vamorolone bone health linear growth vamorolon Duchennova mišična distrofija metabolni zapleti true false true Angleški jezik Slovenski jezik Neznano 2026-01-05 11:23:36 2026-01-05 11:23:37 2026-01-06 03:43:48 0000-00-00 00:00:00 2025 0 0 Nasl. z nasl. zaslona; Opis vira z dne 5. 1. 2025; str. 1-9 Vol. 16 2025 0000-00-00 Zaloznikova Objavljeno NiDoloceno 0000-00-00 0000-00-00 0000-00-00 616-053.2 1664-2392 10.3389/fendo.2025.1697907 263384835 RAZ_Jazbinsek_Soncka_2025.pdf RAZ_Jazbinsek_Soncka_2025.pdf 1 8D0833D9DDA6A3EC2D806B5574DAE032 7e8ee72833fac493c14396856940cd7a437104f2ac305fb14232f54e8742ab82 f95e6e4e-ea20-11f0-94a7-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=37365 https://www.frontiersin.org/journals/endocrinology/articles/10.3389/fendo.2025.1697907 0 080ea346-ea21-11f0-94a7-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=37366 Univerzitetni klinični center Ljubljana 0 0 0